Literature DB >> 23150381

Adeno-associated viral vectors for gene therapy of inherited retinal degenerations.

John G Flannery1, Meike Visel.   

Abstract

Adeno-associated virus (AAV) vectors are in wide use for in vivo gene transfer for the treatment of inherited retinal disease. AAV vectors have been tested in many animal models and have demonstrated efficacy with low toxicity. In this chapter we describe some of the recent methods for small-scale production of these vectors for use in a laboratory setting in volumes and purity appropriate for testing in small and large animals.

Entities:  

Mesh：

Year:  2013        PMID： 23150381     DOI： 10.1007/978-1-62703-080-9_25

Source DB:  PubMed          Journal:  Methods Mol Biol        ISSN： 1064-3745


  13 in total

1.  Optogenetic Retinal Gene Therapy with the Light Gated GPCR Vertebrate Rhodopsin.

Authors:  Benjamin M Gaub; Michael H Berry; Meike Visel; Amy Holt; Ehud Y Isacoff; John G Flannery
Journal:  Methods Mol Biol       Date:  2018

2.  A novel formulation based on 2,3-di(tetradecyloxy)propan-1-amine cationic lipid combined with polysorbate 80 for efficient gene delivery to the retina.

Authors:  Gustavo Puras Ochoa; Jon Zárate Sesma; Mireia Agirre Díez; Ariadna Díaz-Tahoces; Marcelino Avilés-Trigeros; Santiago Grijalvo; Ramón Eritja; Eduardo Fernández; Jose Luis Pedraz
Journal:  Pharm Res       Date:  2014-01-22       Impact factor: 4.200

3.  Amnesia for context fear is caused by widespread disruption of hippocampal activity.

Authors:  Jamie N Krueger; Jacob H Wilmot; Yusuke Teratani-Ota; Kyle R Puhger; Sonya E Nemes; Ana P Crestani; Marrisa M Lafreniere; Brian J Wiltgen
Journal:  Neurobiol Learn Mem       Date:  2020-08-18       Impact factor: 2.877

Review 4.  Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges.

Authors:  Sumit Ghosh; Alex M Brown; Chris Jenkins; Katie Campbell
Journal:  Appl Biosaf       Date:  2020-03-01

5.  Notch Suppression Collaborates with Ascl1 and Lin28 to Unleash a Regenerative Response in Fish Retina, But Not in Mice.

Authors:  Fairouz Elsaeidi; Peter Macpherson; Elizabeth A Mills; Jonathan Jui; John G Flannery; Daniel Goldman
Journal:  J Neurosci       Date:  2018-01-29       Impact factor: 6.167

6.  Neuron-glia signaling in developing retina mediated by neurotransmitter spillover.

Authors:  Juliana M Rosa; Rémi Bos; Georgeann S Sack; Cécile Fortuny; Amit Agarwal; Dwight E Bergles; John G Flannery; Marla B Feller
Journal:  Elife       Date:  2015-08-14       Impact factor: 8.140

7.  NHE8 is essential for RPE cell polarity and photoreceptor survival.

Authors:  Chun-hong Xia; Haiquan Liu; Debra Cheung; Felicia Tang; Bo Chang; Mei Li; Xiaohua Gong
Journal:  Sci Rep       Date:  2015-03-20       Impact factor: 4.379

8.  Specific tools for targeting and expression in Müller glial cells.

Authors:  Lucie P Pellissier; Robert M Hoek; Rogier M Vos; Wendy M Aartsen; Ryan R Klimczak; Stefan A Hoyng; John G Flannery; Jan Wijnholds
Journal:  Mol Ther Methods Clin Dev       Date:  2014-03-19       Impact factor: 6.698

9.  Factors Impacting Efficacy of AAV-Mediated CRISPR-Based Genome Editing for Treatment of Choroidal Neovascularization.

Authors:  Sook Hyun Chung; Iris Natalie Mollhoff; Uyen Nguyen; Amy Nguyen; Natalie Stucka; Eric Tieu; Suman Manna; Ratheesh Kumar Meleppat; Pengfei Zhang; Emerald Lovece Nguyen; Jared Fong; Robert Zawadzki; Glenn Yiu
Journal:  Mol Ther Methods Clin Dev       Date:  2020-01-23       Impact factor: 6.698

10.  Suprachoroidal and Subretinal Injections of AAV Using Transscleral Microneedles for Retinal Gene Delivery in Nonhuman Primates.

Authors:  Glenn Yiu; Sook Hyun Chung; Iris N Mollhoff; Uyen Tu Nguyen; Sara M Thomasy; Jesse Yoo; Donna Taraborelli; Glenn Noronha
Journal:  Mol Ther Methods Clin Dev       Date:  2020-01-21       Impact factor: 6.698
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.